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In spite of the improvements in care and the emergence of disease-modifying treatments, Duchenne muscular dystrophy (DMD) remains a life-limiting disease of adolescence and (young) adulthood. Palliative care approaches and principles should be integrated from the point of diagnosis and implemented throughout the lifespan. A nationwide cross-sectional survey based on a mixed-method-design of qualitative and quantitative research approaches evaluated the structural implementation and perception of palliative care for DMD in Germany. Data analyses revealed that palliative care was predominantly provided at the primary care level by pediatricians, general practitioners and specialized multi-professional outpatient structures. The majority of patients did not utilize the scopes of specialized palliative structures. Simultaneously, insufficiently treated complex symptoms, emergent and elective hospitalizations and barriers in transitioning into adult care presented a considerable burden. A collaborative integrated model with a close cooperation of patients, families and care providers is proposed involving task areas and interfaces complementing primary and specialized palliative care (1) management of complex symptoms, (2) crisis support, (3) intermittent relief of the strain for caregivers, (4) coordination of care, (5) advance care planning and (6) end-of-life care. Specialized palliative care should be used as an "add-on" approach in time of need rather than as a prognosis or disease stage., Competing Interests: Declaration of Competing Interest The authors declared no potential conflicts of interest with respect to research, authorship, and/or publication of this article and received no financial support for the research, authorship, and/or publication of this article. This research did not receive any specific grant from funding agencies in the public, commercial, or non-profit-sectors., (Copyright © 2020 Elsevier B.V. All rights reserved.)

In Germany, every child with a life-limiting condition suffering from symptoms that cannot sufficiently be controlled is eligible by law for specialized pediatric palliative home care (SPPHC). It is the aim of this study to describe the demographic and clinical characteristics of children referred to SPPHC and to compare patients with cancer and non-cancer conditions. The prospective multicenter study includes data on 75 children (median age 7.7 years, 50.7% male). The majority had non-cancer conditions (72%). The most common symptoms were cognitive impairment, somatic pain, impairment in communication or swallowing difficulties. Swallowing difficulties, seizures, and spasticity occurred significantly more often in non-cancer patients ( p < 0.01). Cancer patients received antiemetics significantly more often (permanent and on demand) than non-cancer patients ( p < 0.01). Significantly more non-cancer patients had some type of feeding tube (57.3%) or received oxygen (33.3%) ( p < 0.01). Central venous catheters had been fitted in 20% of the patients, mostly in cancer patients ( p < 0.001). Tracheostomy tubes (9.3%) or ventilation (14.7%) were only used in non-cancer patients. In conclusion, patients referred to SPPHC are a diverse cohort with complex conditions including a large range of neurologically originating symptoms. The care of pediatric palliative care patients with cancer is different to the care of non-cancer patients.

The objective of this prospective study was to assess the prognostic value of dynamic and static liver function tests and clinical symptoms in pediatric patients with chronic end-stage liver disease in a serial examination including three evaluations at 3-month intervals. Of the 24 patients entering the study, six were given transplants within the observation period of 10 months. Of the remaining 18 patients who were considered in the final evaluation, five died before transplantation was possible. The variables included in the analysis were monoethylglycinexylidide (MEGX) formation from lidocaine, bilirubin, albumin, and creatinine serum concentrations, catalytic serum concentration of cholinesterase (CHE), prothrombin time (PT), factors II and V, serum amino acids, body weight, and presence of ascites. In nonsurvivors (n = 5), MEGX serum concentrations 30 min after intravenous administration of lidocaine (1 mg/kg body weight) were < 10 micrograms/L at the first examination. Statistically significant differences between nonsurvivors and survivors were observed for initial MEGX test results (p = 0.0089) and serum bilirubin concentrations (p = 0.009), as well as for the last available MEGX and bilirubin data from each patient (p = 0.017 and 0.016, respectively). At a diagnostic sensitivity of 100%, the corresponding diagnostic specificities for MEGX and bilirubin from the first examination were 77 and 62%, respectively. These data show that consistently low MEGX test results < 10 micrograms/L, obtained 30 min after intravenous administration of lidocaine (1 mg/kg body weight), are a prognostically unfavorable sign in pediatric transplant candidates.